Glascock J, Lenz M, Hobby K, Jarecki J
Cure SMA, Elk Grove Village, Illinois, USA.
Gene Ther. 2017 Sep;24(9):498-500. doi: 10.1038/gt.2017.39. Epub 2017 May 15.
Cure SMA is dedicated to the treatment and cure of spinal muscular atrophy (SMA)-a disease affecting motor neurons, that robs patients of their ability to walk, eat and even breathe. Since 1984, we have directed and invested in comprehensive research that has shaped the scientific community's understanding of SMA. On 23 December, 2016, the Food and Drug Administration (FDA) announced approval of Spinraza, a treatment developed by Biogen and Ionis, making it the first-ever approved therapy for SMA. Cure SMA provided early research funding in 2003 leading to the discovery of ISS-N1 sequence, now targeted by Spinraza. We are pleased that our strategy of providing seed funding for research to either identify new therapeutic strategies or de-risk early stage ones, has proven successful with Spinraza's approval. The approval of Spinraza provides great hope to the SMA community and represents decades of hard work and perseverance by families, researchers, pharmaceutical companies and the FDA. Our hope is that Spinraza is the leading edge of a robust drug pipeline, and with our deep expertise in every aspect of SMA, we remain committed to do everything we can to support research and drug development to achieve the greatest possible effect for each and every SMA patient.
治愈脊髓性肌萎缩症组织致力于脊髓性肌萎缩症（SMA）的治疗与治愈——这是一种影响运动神经元的疾病，会剥夺患者行走、进食甚至呼吸的能力。自1984年以来，我们一直主导并投资开展全面研究，这些研究塑造了科学界对SMA的认知。2016年12月23日，美国食品药品监督管理局（FDA）宣布批准渤健公司和艾奥尼斯公司研发的治疗药物Spinraza，使其成为首个获批用于治疗SMA的疗法。治愈脊髓性肌萎缩症组织在2003年提供了早期研究资金，促成了ISS - N1序列的发现，而Spinraza现在正是针对该序列。我们很高兴，我们为识别新治疗策略或降低早期策略风险的研究提供种子资金的战略，随着Spinraza的获批已被证明是成功的。Spinraza的获批给SMA群体带来了巨大希望，代表着患者家庭、研究人员、制药公司和FDA数十年的辛勤努力与坚持不懈。我们希望Spinraza是强大药物研发线的开端，凭借我们在SMA各个方面的深厚专业知识，我们仍致力于尽一切所能支持研究和药物开发，为每一位SMA患者带来最大可能的疗效。
